Porphyria News and Research

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FDA grants Orphan Drug Designation to Alnylam ALN-AT3 for treatment of hemophilia B

FDA grants Orphan Drug Designation to Alnylam ALN-AT3 for treatment of hemophilia B

Alnylam releases positive top-line data from ALN-TTRsc Phase I trial for TTR-mediated amyloidosis

Alnylam releases positive top-line data from ALN-TTRsc Phase I trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam presents new pre-clinical data of ALN-CC5 for treatment of complement-mediated diseases

Alnylam presents new pre-clinical data of ALN-CC5 for treatment of complement-mediated diseases

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

Alnylam begins dosing in Phase I clinical trial with ALN-TTRsc for treatment of ATTR

Alnylam begins dosing in Phase I clinical trial with ALN-TTRsc for treatment of ATTR

uniQure commences acute intermittent porphyria Phase I clinical trial

uniQure commences acute intermittent porphyria Phase I clinical trial

uniQure receives positive recommendation from EMA CHMP for Glybera

uniQure receives positive recommendation from EMA CHMP for Glybera

Medunik Canada signs exclusive distribution agreement with Orphan Europe

Medunik Canada signs exclusive distribution agreement with Orphan Europe

Gene therapy improves chylomicron metabolism in LPLD patients

Gene therapy improves chylomicron metabolism in LPLD patients

Valeant receives FDA approval for secondary supplier of Methoxsalen

Valeant receives FDA approval for secondary supplier of Methoxsalen

FDA grants orphan designation for AMT's hemophilia gene therapy program

FDA grants orphan designation for AMT's hemophilia gene therapy program

AMT raises € 2.5 million through private placement

AMT raises € 2.5 million through private placement

EMA grants orphan designation to AMT for hemophilia B gene therapy

EMA grants orphan designation to AMT for hemophilia B gene therapy

AMT, NIH enter exclusive license agreement for use of AAV5-based gene therapy vectors

AMT, NIH enter exclusive license agreement for use of AAV5-based gene therapy vectors

AMT total net loss decreases 7% to € 8.7 million for first half year of 2011

AMT total net loss decreases 7% to € 8.7 million for first half year of 2011

Glybera gene therapy reduces pancreatitis risk in Lipoprotein Lipase Deficiency patients

Glybera gene therapy reduces pancreatitis risk in Lipoprotein Lipase Deficiency patients

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

AMT presents long-term efficacy study of Glybera in treatment of lipoprotein lipase deficiency

AMT presents long-term efficacy study of Glybera in treatment of lipoprotein lipase deficiency

Duchenne Parent Project awards grant to AMT for DMD gene therapy

Duchenne Parent Project awards grant to AMT for DMD gene therapy